A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

Официальное название

An Open-label, Multi-centre, Rollover Study to Characterise Long-term Safety and Efficacy of Etavopivat in Adults, Adolescents and Children Who Have Sickle Cell Disease or Thalassaemia and Have Completed a Treatment Period in an Etavopivat Study

Обзор

Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin. Haemoglobin is the protein that carries oxygen through the body. This study is looking into how safe treatment with etavopivat is and how well it works over a long period of time. The study will last for up to 264 weeks, but it will end earlier if etavopivat is approved in the participant's country.

Вмешательства

• Препарат Etavopivat A
  Participants will receive an oral dose of Etavopivat A.
• Препарат Etavopivat B
  Participants will receive an oral dose of Etavopivat B.
• Препарат Etavopivat C
  Participants will receive an oral dose of Etavopivat C.

Первичные конечные точки

• Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately [Срок оценки: Baseline (week 0 of FLORAL) up to end of study (up to week 316)]
• Number of adverse reactions, reported for each indication and age group separately [Срок оценки: Baseline (week 0 of FLORAL) up to end of study (up to week 316)]

Вторичные конечные точки (8)

• Annualised vaso-occlusive crisis (VOC) rates, reported for each age group separately [Срок оценки: Baseline (week 0 of FLORAL) up to end of treatment (up to week 312)]
• Change in VOCs, reported for each age group separately [Срок оценки: Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312)]
• Change in hemoglobin (Hb) concentration, reported for each age group separately [Срок оценки: Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312)]
• Annualised number of hospitalisations, reported for each age group separately [Срок оценки: Baseline (week 0 of FLORAL) up to end of treatment (up to week 312)]
• Average length of stay of hospitalisations, reported for each age group separately [Срок оценки: Baseline (week 0 of FLORAL) up to end of treatment (up to week 312)]
• Change in Hb concentration [Срок оценки: Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312)]
• Number of red blood cell (RBC) units transfused, reported for each indication separately [Срок оценки: Baseline (week 0 of FLORAL) up to end of treatment (up to week 312)]
• Change in RBC units transfused, reported for each indication separately [Срок оценки: Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312)]

Критерии участия

Критерии включения

• Participant must have ongoing participation in an etavopivat parent study for treatment of sickle cell disease (SCD) or thalassaemia and have completed at least a treatment period of the parent study.
• Participant must have derived clinical benefit from treatment with etavopivat, as determined by the investigator.
• Any participant with dose reduction or temporary discontinuation will need to be successfully rechallenged to the full dose of etavopivat before transferring.
• Participants on hydroxyurea (HU), crizanlizumab or l-glutamine oral powder (Endari®) treatment at the time of consent may be eligible if they have been on a stable dose in the parent study as defined at the investigator's discretion. Necessary adjustments related to weight or age are accepted. Participants with temporary dose reductions or pauses due to medical reasons may still be considered to have a stable dose, as determined by the investigator, who will assess the impact of these adjustments based on clinical context and the participant's overall health status.

Критерии исключения

• Any disorder, except for conditions associated with SCD or thalassaemia, which in the investigator's opinion might jeopardise participant's safety or compliance with the protocol.
• Participant withdrew or had permanent treatment discontinuation from an etavopivat clinical study.
• Participants on permanent dose reduction (greater than \[>\] 28 days or more) or ongoing temporary treatment discontinuation.
• Use of any of the following within the timeframes prior to the transfer visit as stated:
• Use of haemoglobin S (HbS) polymerisation inhibitors within participation of the parent study or anticipated need for this agent during this study.
• Use of an experimental selectin antagonist (e.g., monoclonal antibody or small molecule) within the parent study or anticipated need for such agents during this study.
• Use of erythropoietin or other haematopoietic growth factor treatment for more than 4 consecutive weeks during the parent study or anticipated need of such agents for a maintenance treatment during this study.
• Receiving or use of concomitant medications that are strong inducers of cytochrome P450 (CYP) 3A4 within 2 weeks of the transfer visit or anticipated need for such agents during the study.
• Current participation in a study that is not a designated parent study, or planned participation in any other clinical study, for the duration of FLORAL.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 39 центров

• Univ of Alabama Birmingham — Birmingham
• Phoenix Children's Hsptl — Phoenix
• Children's Hospital Los Angeles - Endocrinology — Los Angeles
• Children's Hospital Los Angeles - Endocrinology — Los Angeles
• UCSF Oakland Benioff ChildHosp — Oakland
• UCSF Oakland Benioff ChildHosp — Oakland
• Children's Hosp Of Orange — Orange
• University Of California Irvine — Orange
• … и ещё 31 центр

Великобритания · 8 центров

Список центров уточняется — проверьте первичный протокол.

Turkey (Türkiye) · 7 центров

Список центров уточняется — проверьте первичный протокол.

Индия · 6 центров

• All India Institute of Medical Sciences (AIIMS), Raipur — Raipur
• K.J Somaiya Hospital and Research Centre — Mumbai
• Victoria Hospital, Bangalore — Bangalore
• Nirmal Hospital Pvt. Ltd. — Gujarat
• Suretech Hospital and Research Centre Ltd. — Maharashtra
• All India Institute of Medical Sciences_Delhi — New Delhi

Kenya · 6 центров

• Gertrude's Children's Hospital — Nairobi
• KEMRI CRDR Siaya Clinical Research Annex, Country Referral Hospital — Siaya
• KEMRI-Walter-Reed Kericho — Kericho
• Kombewa Clinical Research Centre — Kisumu
• KEMRI Kondele Children Hospital, Kisumu — Kisumu
• Ahero Clinical Trials Unit — Kisumu

Nigeria · 6 центров

• University of Nigeria Teaching Hospital (UNTH) — Ituku-Ozalla
• University College Hospital Paediatric Haematology and Oncology Unit, Ibadan — Ibadan
• … и ещё 4 центра

Египет · 5 центров

• Alexandria University Hospital — Alexandria, Egypt
• Zagazig University Hospital — Alsharkia, Egypt
• Faculty of Medicine Ain Shams Medical Research Institute (MASRI) — Cairo
• Cairo University — Cairo, Egypt
• Abu El-Reesh El-Mounira Children University Hospital — Cairo, Egypt

Канада · 4 центра

• St Pauls Hospital — Vancouver
• The Hospital for Sick Children — Toronto
• University Health Network - Toronto General Hospital — Toronto
• CHU Ste-Justine — Montreal

Франция · 4 центра

• Ap-Hp-Hopital Henri Mondor — Créteil
• Hospices Civils de Lyon-Hopital Edouard Herriot — Lyon
• Ap-Hp-Hopital Robert Debre — Paris
• Ap-Hp-Hopital Robert Debre — Paris

Греция · 4 центра

• General Hospital Of Larissa Koutlibaneio And Triantafylleio - Thalassemia and SCD Unit — Larissa
• Hippokration Hospital — Athens
• General University Hospital of Patras — Pátrai
• 'Ippokrateio' General Hospital of Thessaloniki — Thessaloniki

Италия · 3 центра

• Azienda Ospedaliera Universitaria San Luigi Gonzaga - S.C.D.O. Microcitemie e malattie rar — Orbassano
• Azienda Ospedale Universita Padova — Padova
• Fondazione IRCCS Policlinico San Matteo — Pavia

Ливан · 3 центра

• American University of Beirut Medical Centre — Hamra
• Chronic Care Center — Hazmiyeh
• Hospital Nini — Tripoli

Испания · 3 центра

Список центров уточняется — проверьте первичный протокол.

Германия · 2 центра

• Charité - Campus Virchow-Klinikum - Klinik für Pädiatrie mit Schwerpunkt Onkologie und Häm — Berlin
• Universitätsklinikum Freiburg - Kinder- und Jugendklinik — Freiburg im Breisgau

Ghana · 2 центра

• Kintampo Health Research Centre (KHRC) — Kintampo
• Ghana Institute of Clinical Genetics, Korle Bu Teaching Hospital (KBTH) — Accra

Саудовская Аравия · 2 центра

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Oman · 1 центр

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Идентификаторы

NCT: NCT06609226 · NN7535-7822 · U1111-1301-8130 · 2024-510805-27

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗